Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cells

β-thalassemias (β-thal) are a group of blood disorders caused by mutations in the β-globin gene (HBB) cluster. β-globin associates with α-globin to form adult hemoglobin (HbA, α(2)β(2)), the main oxygen-carrier in erythrocytes. When β-globin chains are absent or limiting, free α-globins precipitate...

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Veröffentlicht in:Blood Adv
Hauptverfasser: Pavani, Giulia, Fabiano, Anna, Laurent, Marine, Amor, Fatima, Cantelli, Erika, Chalumeau, Anne, Maule, Giulia, Tachtsidi, Alexandra, Concordet, Jean-Paul, Cereseto, Anna, Mavilio, Fulvio, Ferrari, Giuliana, Miccio, Annarita, Amendola, Mario
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Society of Hematology 2021
Schlagworte:
Gene Therapy
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7948300/
https://ncbi.nlm.nih.gov/pubmed/33635334
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/bloodadvances.2020001996
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